Gene Augmentation for X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR

X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene is a severe and early onset form of retinal degeneration, and no treatment is currently available. Recent evidence in two clinically relevant canine models shows that adeno-associated viral (AAV)-mediated RPGR gene transfer to...

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Pubblicato in:Cold Spring Harb Perspect Med
Autori principali: Beltran, William A., Cideciyan, Artur V., Lewin, Alfred S., Hauswirth, William W., Jacobson, Samuel G., Aguirre, Gustavo D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Cold Spring Harbor Laboratory Press 2015
Soggetti:
Perspectives
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4315918/
https://ncbi.nlm.nih.gov/pubmed/25301933
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/cshperspect.a017392
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